A Randomized Phase 1 of of MZE001 in Healthy Volunteers

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study of MZE001 to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Healthy Subjects

This is a Phase 1, randomized, double-blind, placebo-controlled, single (SAD) / multiple (MAD) ascending dose and food effect study.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: MZE001; Drug: Placebo

Detailed Description

Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered MZE001 in healthy subjects, in fasted and fed states. Each SAD and MAD cohort will enroll 8 subjects randomized 6 active: 2 placebo to receive single or multiple doses of MZE001.

• Study Type: Interventional
• Enrollment (Actual): 121
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Tustin, California, United States, 92780
      • Orange County Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Male and female subjects, ages 18 - 55 years, inclusive;
2. Must provide written informed consent prior to any study assessments and be willing and able to comply with all study procedures;
3. Body mass index (BMI) between 18.0 and 32.0 kg/m2 inclusive;
4. Healthy as determined by the Investigator based on pre-study medical history, physical examination, and baseline safety laboratory studies.
5. Able to complete exercise treadmill test with no cardiac abnormalities detected
6. Females of childbearing potential who are sexually active with a non-sterilized partner must use a highly effective method of contraception during study participation and for 30 days after last administration of study drug.
7. Males of childbearing potential must use a highly effective method of birth control during study participation and for 90 days after last administration of study drug.

Exclusion Criteria:

1. Any concurrent condition that in the opinion of the Investigator would interfere with the evaluation of the investigational product or lead to increased risk of harm;
2. Any history of coronary artery disease or cardiovascular disease;
3. History or presence of gastrointestinal, renal, or hepatic disease or any other condition known to interfere with absorption, distribution, metabolism, or excretion of drugs;
4. History of cancer within past 5 years, with the exception of non-melanoma skin cancer and treated / excised melanoma;
5. Any clinically important illness, medical/surgical procedure, or trauma within 4 weeks prior to the Screening visit;
6. Fridericia's corrected QT (QTcF) > 450 ms for male participants and > 470 ms for female participants or history of QT interval prolongation;
7. History or presence of an abnormal ECG that is clinically significant in the Investigator's opinion
8. Resting seated blood pressure < 90/40 mmHg or > 140/90 mmHg
9. Resting seated heart rate < 45 bpm or > 99 bpm
10. Poor peripheral venous access;
11. Have a history of drug hypersensitivity or anaphylaxis;
12. Current smoker or recent history of smoking within the last 3 months prior to the Screening visit;
13. Have a history of alcoholism or drug abuse or positive drug screen
14. Have used any prescription or non-prescription medicines or have been administered a vaccine within 14 days of admission,
15. Have received any investigational drug within 30 days or < 5 half-lives, whichever is longer, prior to the Screening visit;
16. Have donated or received any blood or blood products within the 3 months prior to the Screening visit.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: SEQUENTIAL
• Masking: TRIPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: MZE001; MZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease.	Drug: MZE001; Small molecule inhibitor of muscle glycogen synthase; Other Names: MZ1449348
PLACEBO_COMPARATOR: Placebo; Excipients containing no active ingredients.	Drug: Placebo; Product containing excipients with no active ingredients; Other Names: mannitol, silicified microcrystalline cellulose, magnesium stearate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse events as a measure of safety and tolerability of MZE001	Occurrence of adverse events, serious adverse events, adverse events of special interest	14 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum concentration following multiple doses of MZE001	PK	14 days
AUC following multiple doses of MZE001	PK	14 days
Accumulation ratio following multiple doses of MZE001	PK	14 days

Collaborators and Investigators

• Sponsor: Maze Therapeutics

Study record dates

Study Major Dates

• Study Start (ACTUAL): February 7, 2022
• Primary Completion (ACTUAL): December 21, 2022
• Study Completion (ACTUAL): December 21, 2022

Study Registration Dates

• First Submitted: February 8, 2022
• First Submitted That Met QC Criteria: February 17, 2022
• First Posted (ACTUAL): February 21, 2022

Study Record Updates

• Last Update Posted (ACTUAL): February 6, 2023
• Last Update Submitted That Met QC Criteria: February 2, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Physiological Effects of Drugs; Natriuretic Agents; Diuretics, Osmotic; Diuretics; Mannitol
• Other Study ID Numbers: MZE001-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No